The FDA’s decision was not unexpected, as the proprietary extract formulation — developed by the British biotechnology firm GW Pharmaceuticals — had previously demonstrated safety and clinical efficacy at reducing seizure frequency in several placebo-controlled trials. Epidiolex had previously received Fast Track Designation and Orphan Drug Status from the FDA. It is the fourth marijuana-based medicine to receive US FDA approval — joining dronabinol (aka Marinol), nabilone (aka Cesamet), and liquid synthetic THC (aka Syndros). However, Epidiolex is the first FDA-approved medicine containing plant-derived, non-synthetic cannabinoids.

Commenting on the agency’s decision, NORML Deputy Director Paul Armentano said: “The FDA’s approval of this plant-derived medicine provides an additional option to patients seeking the therapeutic benefits of cannabis. However, it remains to be seen whether physicians will be comfortable prescribing this new agent to those patients who may benefit from it, and whether it will be priced in a range that patients may afford.” According to the New York Times, analysts expect Epidiolex to cost $2,500 to $5,000 a month.

He added: “We anticipated that Epidiolex will be the first of many potential FDA-approved medicines based on the cannabis plant. Nonetheless, these alternatives should not be regulated as options to replace the use and regulation of herbal cannabis — a product that humans have used safely and effectively as a medicine for thousands of years and is approved today by statute in 30 states.”

Federal agencies have 90 days to determine the scheduling of Epidiolex. The new drug is anticipated to become available to patients later this fall. In clinical trials, patients administered Epidiolex, on average, obtained a 40 percent reduction in seizure frequency.

Lennox-Gaustaut syndrome is estimated to account for between one and four percent of all cases of childhood epilepsy. Dravet syndrome is estimated to effect about 1 in 40,000 people.

Despite today’s approval, the FDA acknowledged in a statement that the cannabidinoid CBD still remains classified at this time as a schedule I controlled substance, and that the agency is “prepared to take action when we see the illegal marketing of CBD-containing products with serious, unproven medical claims.” FDA Commissioner Scott Gottlieb further added: “This is the approval of one specific CBD medication for a specific use. … [T]his is not an approval of marijuana or all of its components.”

The administration of oral cannabis extracts is associated with the mitigation of seizures in adolescents with epilepsy, according to clinical data published this month in the journal Epilepsy & Behavior.

Researchers from the Colorado Children’s Hospital in Denver performed a retrospective chart review of 75 children provided cannabis extracts. Authors reported that 57 percent of subjects showed some level of improvement in seizure control while 33 percent reported a greater than 50 percent reduction in seizure frequency.

Researchers also reported “improved behavior/alertness” in one-third of subjects and improved motor skills in ten percent of treated patients. Adverse events were reported in 44 percent of subjects, 13 percent of which reported increased seizure activity. Overall, however, authors concluded that the extracts were “well tolerated by children.”

Separate clinical trial results publicized last week at the 67th Annual Meeting of the American Academy of Neurology reported that the administration of a proprietary form of CBD (cannabidiol) extracts decreased seizure frequency by 54 percent over a 12-week period in children with treatment-resistant epilepsy.

Survey data compiled by Stanford University in 2013 reported that the administration of cannabidiol-enriched cannabis decreased seizures in 16 of 19 patients with pediatric epilepsy.

Last February, the Epilepsy Foundation of America enacted a resolution in support of the “rights of patients and families living with seizures and epilepsy to access physician directed care, including medical marijuana.”

An abstract of the study, “Parental reporting of response to oral cannabis extracts for treatment of refractory epilepsy,” appears online here.

Cananbidiol (CBD), a non-psychotropic cannabinoid, alleviates psychotic symptoms and may hold promise as an alternative antipsychotic treatment, according to a review published in the November issue of the journal Neuropsychopharmacology.

Investigators in the Netherlands and in the United Kingdom reviewed preclinical and clinical data on the use of CBD as an antipsychotic agent. Authors reported that both animal and human studies document the ability of CBD to mitigate symptoms of psychosis. Specifically, CBD administration is associated with improved symptoms in clinical evaluations of patients with schizophrenia, Parkinson’s disease, and ketamine-induced dissociative and psychotic symptoms.

Investigators also highlighted a 2012 double-blind, randomized placebo-controlled trial assessing CBD versus the prescription anti-psychotic drug amisulpride in 42 subjects with schizophrenia and acute paranoia. Authors reported that both CBD and the prescription drug were associated with “equally significant clinical improvement” in this patient population, but that cannabidiol “possessed significantly less side effects.”

Researchers concluded: “[E]vidence from several study domains suggests that CBD has some potential as an antipsychotic treatment. … Given the high tolerability and superior cost-effectiveness, CBD may prove to be an attractive alternative to current antipsychotic treatment.”

Previous human trials assessing the administration of CBD in healthy human subjects report that the cannabinoid is “safe and well tolerated.”

Separate investigations of CBD, primarily in animal models, have documented the cannabinoid to possess a variety of therapeutic qualities, including anti-inflammatory, anti-diabetic, anti-epileptic, anti-cancer, and bone-stimulating properties. Recently, the FDA approved the experimental use of CBD extracts for the treatment of a rare form of intractable pediatric epilepsy known as Dravet syndrome. Preliminary clinical trials assessing the safety and tolerability of the compound in children are scheduled to begin imminently.
Full text of the study, entitled “Cannabidiol as a potential treatment for psychosis,” appears online here.